EP2609198B8 - SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER - Google Patents
SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER Download PDFInfo
- Publication number
- EP2609198B8 EP2609198B8 EP11820420.5A EP11820420A EP2609198B8 EP 2609198 B8 EP2609198 B8 EP 2609198B8 EP 11820420 A EP11820420 A EP 11820420A EP 2609198 B8 EP2609198 B8 EP 2609198B8
- Authority
- EP
- European Patent Office
- Prior art keywords
- internal
- nucleic acid
- agents containing
- rnai agents
- stranded rnai
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
- 108091030071 RNAI Proteins 0.000 title 1
- 239000003795 chemical substances by application Substances 0.000 title 1
- 230000009368 gene silencing by RNA Effects 0.000 title 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3183—Diol linkers, e.g. glycols or propanediols
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Priority Applications (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP18000072.1A EP3372684B1 (en) | 2010-08-24 | 2011-08-19 | Single-stranded rnai agents containing an internal, non-nucleic acid spacer |
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US37647110P | 2010-08-24 | 2010-08-24 | |
PCT/US2011/048338 WO2012027206A1 (en) | 2010-08-24 | 2011-08-19 | SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER |
Related Child Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EP18000072.1A Division EP3372684B1 (en) | 2010-08-24 | 2011-08-19 | Single-stranded rnai agents containing an internal, non-nucleic acid spacer |
EP18000072.1A Division-Into EP3372684B1 (en) | 2010-08-24 | 2011-08-19 | Single-stranded rnai agents containing an internal, non-nucleic acid spacer |
Publications (4)
Publication Number | Publication Date |
---|---|
EP2609198A1 EP2609198A1 (en) | 2013-07-03 |
EP2609198A4 EP2609198A4 (en) | 2014-11-19 |
EP2609198B1 EP2609198B1 (en) | 2018-02-21 |
EP2609198B8 true EP2609198B8 (en) | 2018-03-28 |
Family
ID=45723747
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EP18000072.1A Active EP3372684B1 (en) | 2010-08-24 | 2011-08-19 | Single-stranded rnai agents containing an internal, non-nucleic acid spacer |
EP11820420.5A Active EP2609198B8 (en) | 2010-08-24 | 2011-08-19 | SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EP18000072.1A Active EP3372684B1 (en) | 2010-08-24 | 2011-08-19 | Single-stranded rnai agents containing an internal, non-nucleic acid spacer |
Country Status (6)
Country | Link |
---|---|
US (3) | US9243246B2 (ko) |
EP (2) | EP3372684B1 (ko) |
JP (5) | JP6106085B2 (ko) |
KR (1) | KR20130137160A (ko) |
CN (1) | CN103140582A (ko) |
WO (1) | WO2012027206A1 (ko) |
Families Citing this family (36)
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US9657294B2 (en) | 2002-02-20 | 2017-05-23 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
US9181551B2 (en) | 2002-02-20 | 2015-11-10 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
US8748405B2 (en) * | 2007-01-26 | 2014-06-10 | City Of Hope | Methods and compositions for the treatment of cancer or other diseases |
WO2011028550A1 (en) | 2009-08-24 | 2011-03-10 | Merck Sharp & Dohme Corp. | Segmented micro rna mimetics |
US9243246B2 (en) * | 2010-08-24 | 2016-01-26 | Sirna Therapeutics, Inc. | Single-stranded RNAi agents containing an internal, non-nucleic acid spacer |
EP3766975A1 (en) | 2010-10-29 | 2021-01-20 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of gene expression using short interfering nucleic acid (sina) |
CA2817371A1 (en) | 2010-12-15 | 2012-06-21 | Miragen Therapeutics | Microrna inhibitors comprising locked nucleotides |
KR20140091688A (ko) | 2011-10-06 | 2014-07-22 | 미라젠 세러퓨틱스 인코포레이티드 | 마이크로rna 조절에 의한 전신 에너지 항상성의 제어 |
WO2013165816A2 (en) | 2012-05-02 | 2013-11-07 | Merck Sharp & Dohme Corp. | SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS |
US9334498B2 (en) | 2012-05-10 | 2016-05-10 | Uab Research Foundation | Methods and compositions for modulating MIR-204 activity |
US20140378526A1 (en) * | 2012-05-11 | 2014-12-25 | City Of Hope | Design of nucleic acid binding molecules with non-watson crick and non-canonical pairing based on artificial mutation consensus sequences to counter escape mutations |
US9512163B2 (en) * | 2012-05-21 | 2016-12-06 | Agilent Technologies, Inc. | Compositions and methods for conjugating oligonucleotides |
US20150140591A1 (en) * | 2012-06-06 | 2015-05-21 | Boehringer Ingelheim International Gmbh | CELL ENGINEERING USING RNAs |
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CN104560992B (zh) * | 2013-10-10 | 2018-01-02 | 复旦大学附属妇产科医院 | 一种用于预防和治疗人乳头瘤病毒感染和宫颈癌的微小rna |
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CN103924002B (zh) * | 2014-05-04 | 2016-01-20 | 山东大学 | 血清中的microRNA作为肝癌诊断标志物的应用 |
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JP2018503646A (ja) | 2015-01-20 | 2018-02-08 | ミラゲン セラピューティクス, インコーポレイテッド | miR−92阻害剤およびその使用 |
ES2876930T3 (es) | 2015-08-20 | 2021-11-15 | Asociacion Centro De Investig Cooperativa En Biociencias Cic Biogune | Métodos y composiciones para tratar enfermedades y afecciones hepáticas |
TWI685500B (zh) * | 2015-10-13 | 2020-02-21 | 長庚大學 | 以微核酸-520b(miR-520b)序列作爲抑制頭頸癌腫瘤生長、侵犯與轉移及其醫藥組成物之用途 |
AU2016365828A1 (en) * | 2015-12-08 | 2018-07-05 | Solstice Biologics, Ltd. | Polynucleotide constructs having an auxiliary moiety non-bioreversibly linked to an internucleoside phosphate or phosphorothioate |
WO2017181088A1 (en) * | 2016-04-14 | 2017-10-19 | University Of Florida Research Foundation, Incorporated | Use of mir-223-3p as a cancer therapeutic and method for treating cancer using the same |
CN105861728A (zh) * | 2016-06-12 | 2016-08-17 | 上海市第十人民医院 | 循环miRNA作为年龄相关黄斑变性诊断标志物中的应用 |
CN107630015B (zh) * | 2016-07-19 | 2021-03-09 | 上海市东方医院 | 一种稳定的dna-rna双链结构 |
JP2019532027A (ja) | 2016-08-17 | 2019-11-07 | ソルスティス バイオロジクス,リミティッド | ポリヌクレオチド構築物 |
JP2019535839A (ja) | 2016-11-29 | 2019-12-12 | ピュアテック ヘルス エルエルシー | 治療剤の送達のためのエクソソーム |
EP3645546A4 (en) | 2017-06-30 | 2021-12-01 | Solstice Biologics, Ltd. | CHIRAL PHOSPHORAMIDITIS AUXILIARIES AND THEIR METHODS OF USE |
JP7162918B2 (ja) * | 2018-05-31 | 2022-10-31 | コリア ユニバーシティ リサーチ アンド ビジネス ファウンデーション | マイクロrnaを抑制する変形核酸及びその用途 |
JP7378739B2 (ja) | 2018-08-10 | 2023-11-14 | 東レ株式会社 | 前立腺がんの検出のためのキット、デバイス及び方法 |
CN109706239A (zh) * | 2018-09-26 | 2019-05-03 | 南京市妇幼保健院 | 一种miR-1976在防治细菌性阴道病中的应用 |
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JP2012517226A (ja) * | 2009-02-10 | 2012-08-02 | イデラ ファーマシューティカルズ インコーポレイテッド | Tlr7の合成rnaベースのアゴニスト |
CA2753975C (en) * | 2009-03-02 | 2017-09-26 | Alnylam Pharmaceuticals, Inc. | Nucleic acid chemical modifications |
WO2011028550A1 (en) * | 2009-08-24 | 2011-03-10 | Merck Sharp & Dohme Corp. | Segmented micro rna mimetics |
HUE026020T2 (en) * | 2009-08-27 | 2016-05-30 | Idera Pharmaceuticals Inc | Preparation for inhibiting gene expression and its uses |
US10913767B2 (en) * | 2010-04-22 | 2021-02-09 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides comprising acyclic and abasic nucleosides and analogs |
US9243246B2 (en) * | 2010-08-24 | 2016-01-26 | Sirna Therapeutics, Inc. | Single-stranded RNAi agents containing an internal, non-nucleic acid spacer |
WO2012068187A1 (en) | 2010-11-19 | 2012-05-24 | Merck Sharp & Dohme Corp. | Poly(amide) polymers for the delivery of oligonucleotides |
DK3366775T3 (da) | 2011-11-18 | 2022-08-01 | Alnylam Pharmaceuticals Inc | Modificerede rnai-midler |
WO2013165816A2 (en) | 2012-05-02 | 2013-11-07 | Merck Sharp & Dohme Corp. | SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS |
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2011
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- 2011-08-19 EP EP11820420.5A patent/EP2609198B8/en active Active
- 2011-08-19 KR KR1020137007266A patent/KR20130137160A/ko not_active Application Discontinuation
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US20160222381A1 (en) | 2016-08-04 |
EP2609198A4 (en) | 2014-11-19 |
JP2022037070A (ja) | 2022-03-08 |
US9845466B2 (en) | 2017-12-19 |
JP6106085B2 (ja) | 2017-03-29 |
EP3372684B1 (en) | 2020-10-07 |
EP2609198A1 (en) | 2013-07-03 |
JP2013535982A (ja) | 2013-09-19 |
JP2023100832A (ja) | 2023-07-19 |
CN103140582A (zh) | 2013-06-05 |
US9243246B2 (en) | 2016-01-26 |
US20180080024A1 (en) | 2018-03-22 |
US20130171242A1 (en) | 2013-07-04 |
EP2609198B1 (en) | 2018-02-21 |
JP2020074788A (ja) | 2020-05-21 |
WO2012027206A1 (en) | 2012-03-01 |
JP2017079776A (ja) | 2017-05-18 |
US10584335B2 (en) | 2020-03-10 |
EP3372684A1 (en) | 2018-09-12 |
JP7527437B2 (ja) | 2024-08-02 |
KR20130137160A (ko) | 2013-12-16 |
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